Background. The key goal of autologous hematopoietic stem cell transplantation (auto-HSCT) is to overcome bone marrow failure, which complicates high-dose chemotherapy. In 2024, 2,790 HSCTs were performed in Russia, 61 % of which were autologous and 39 % allogeneic. Transplant activity in the country has increased by 46 % over the past 5 years. Aim. To evaluate the immediate and long-term outcomes of auto-HSCT in patients with hematologic malignancies treated over the past 6 years (2020–2025) in the High-Dose Chemotherapy Department with the Bone Marrow Transplantation Unit at the P. A. Herzen Moscow Oncology Research Institute. Materials and methods. This retrospective observational study included data from 187 patients who underwent auto-HSCT at the P. A. Herzen Moscow Oncology Research Institute. For the analysis, patients were divided into 3 nosologic groups: multiple myeloma (MM) – 127 (68 %), non-Hodgkin’s lymphoma (NHL) – 31 (16.6 %), and classical Hodgkin’s lymphoma (cHL) – 29 (15.4 %) patients. The median age ranged from 33 to 59 years depending on the nosology. The study assessed the parameters of CD34+ cell mobilization, conditioning regimens, hematological toxicity, overall survival (OS), and progression-free survival (PFS). Survival analysis was performed using the Kaplan–Meier method. Results. A total of 203 auto-HSCT procedures were performed, including 16 (7.9 %) tandem transplants for MM. The median of collected CD34+ cells was 8.1 × 106 / kg in MM, 9.4 × 106 / kg in cHL, and 7.6 × 106 / kg in NHL. Hematopoiesis was restored within comparable time frames in all groups (median agranulocytosis duration was 9–12 days). With a median follow-up of 20–25 months, the 2-year PFS rates were 91.6 % (MM), 92.0 % (cHL), and 81.6 % (NHL); 2-year OS was 96.3 % (MM), 100 % (cHL), and 85.6 % (NHL). Patients with MM showed a significant improvement in the response after transplantation (the proportion of complete responses increased from 9.4 to 61.4 %; p = 0.003). Conclusion. The results of our single-center study demonstrate the high efficacy and acceptable safety profile of auto-HSCT, comparable to global data. Further development of high-dose therapy programs, optimization of mobilization, and the introduction of new maintenance therapy regimens are promising areas for improving treatment outcomes for patients with hematological malignancies in the Russian Federation as a whole.
Background. Infectious complications remain a leading cause of early transplant-related mortality following allogeneic hematopoietic stem cell transplantation (allo-HSCT), particularly during the period of febrile neutropenia. Despite advances in transplantation techniques and supportive care, the optimal strategy for empirical antibacterial therapy in this high-risk population remains controversial, especially in resource-limited settings. Aim. To evaluate the incidence and spectrum of infectious complications, their impact on early mortality after allo-HSCT, and to assess the effectiveness and safety of an escalation-based empirical antibacterial therapy strategy in a single transplantation center with limited resources. Materials and methods. A single-center retrospective study included 45 adult patients who underwent allo-HSCT between 2023 and 2025 (acute myeloid leukemia – 21, acute lymphoblastic leukemia – 15, aplastic anemia – 8, myelodysplastic syndrome – 1). All patients received a uniform conditioning regimen based on fludarabine and melphalan (FluMel140). The incidence of febrile neutropenia, bloodstream infections, sepsis and septic shock, intensive care unit admission, and early mortality (≤30 days post-transplant) were analyzed. All patients received empirical antibacterial therapy following an escalation strategy. Microbiological identification was performed using VITEK 2 COMPACT and VITEK MS PRIME systems. Results. Febrile neutropenia occurred in 100 % of patients. Recurrent febrile neutropenia was observed in 44 % of cases, requiring escalation of antibacterial therapy to carbapenems. Microbiologically documented bloodstream infections were identified in 13 % of patients. Sepsis with subsequent septic shock developed in 22 % of patients and was associated with 100 % mortality in this subgroup. Gram-negative pathogens predominated among fatal infections, with Klebsiella pneumoniae and Pseudomonas aeruginosa being the most frequently identified organisms. Late colonization with Klebsiella pneumoniae was observed in all patients who died from infectious complications. Conclusion. Infectious complications remain the principal cause of early mortality after allo-HSCT. An escalation-based empirical antibacterial therapy strategy represents a feasible and reproducible approach in the majority of allo-HSCT recipients. However, in patients with a high infectious risk profile and colonization with multidrug-resistant organisms, this strategy may be insufficient. The obtained results highlight the need for individualized empirical therapy, enhanced microbiological monitoring, and optimization of conditioning regimens, particularly in resource-limited transplant centers.
Background. Thrombopoietin receptor agonists are considered second-line therapy and are prescribed to patients with insufficient response or intolerance to first-line therapy. These drugs, which stimulate platelet production by activating the thrombopoietin receptor, are effective and safe for both short-term and long-term treatment of immune thrombocytopenia. They provide high response rates in 70–80 % of patients and sustained remission in 10–30 %. The response to long-term use of thrombopoietin receptor agonists lasts for 6–8 years or more and allows for the reduction or discontinuation of corticosteroid and immunosuppressant use, as well as a halving of the incidence of bleeding. Aim. To evaluate the efficacy of the thrombopoietin receptor agonist avatrombopag in patients with immune thrombocytopenia. Materials and methods. The study included 45 patients with immune thrombocytopenia who received avatrombopag orally once daily, starting with a dose of 20 mg and subsequently titrated to achieve and maintain a platelet count of at least 50 × 109 / L. Statistical data were processed using GraphPad Prism 9, and differences between pre- and post-therapy were assessed using the Wilcoxon signed-rank test (p 0.05). Results. The results of the study demonstrate a statistically significant increase in platelet counts among patients with immune thrombocytopenia treated with avatrombopag. Conclusion. The use of avatrombopag appears justified as a second-line treatment for immune thrombocytopenia.
Aim. To evaluate the structure and effectiveness of therapy for newly diagnosed diffuse large B-cell lymphoma (DLBCL) in adults in real clinical practice according to the Moscow Oncology Registry data. Materials and methods. Data from 1060 patients with newly diagnosed DLBCL from the Moscow Oncology Registry who received treatment from 2022 to 2025 in a Moscow healthcare setting were retrospectively analyzed. The median age was 69 years. The majority of patients were in the older age group (60.4 %), with advanced disease stages (70 %), non-GCB subtypes (60 %), and high risk according to the international prognostic index (67 %). Of all patients included, 62.1 % received immunochemotherapy according to the R-CHOP program (rituximab, cyclophosphamide, doxorubicin, vincristine, prednisolone), 12.6 % received R-DA-EPOCH (rituximab, etoposide, prednisolone, vincristine, cyclophosphamide, doxorubicin), 2.6 % received intensive regimens, 14.7 % received R-miniCHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, prednisolone in reduced doses), 7.8 % received RB (rituximab, bendamustine) or R-CVP (rituximab, cyclophosphamide, vincristine, prednisolone). Results. In the overall patient cohort (n = 1060), the objective response rate was 79.7 % (n = 845). Complete metabolic response was achieved by 66.4 % (n = 704) of patients. The complete response rate varied depending on the treatment regimen: in the R-CHOP group it was 68.4 % (n = 451), when using R-DA-EPOCH – 69.4 % (n = 93), intensive regimens – 89.3 % (n = 25). Among patients receiving R-miniCHOP, complete response was achieved in 62.2 % (n = 97) of cases, while in the RB / R-CVP group this rate was the lowest and amounted to 45.8 % (n = 38). With a median follow-up of 24 months, the 2-year overall survival in the total group was 70 % (95 % confidence interval 67–73), and the 2-year progression-free survival was 44 % (95 % confidence interval 40–48). Conclusion. Results of first-line DLBCL therapy efficacy analysis indicate unsatisfactory treatment outcomes, particularly in the high-risk group for early progression. We suggest that biologically targeted antitumor therapy based on the identified tumor genotype represents the most promising clinical approach for patients with newly diagnosed DLBCL.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal hematologic disorder whose pathogenesis is primarily driven by the formation of a cell clone (the PNH clone) lacking surface protective glycoproteins (CD55 and CD59), activation of the alternative complement pathway, and complement-mediated destruction of erythrocytes within blood vessels (intravascular hemolysis). This process underlies the main clinical manifestations of PNH – hemolytic anemia and thrombotic complications. The introduction of eculizumab, a C5 complement inhibitor, into clinical practice in 2007, which blocks the mechanism of intravascular hemolysis, revolutionized the course and prognosis of PNH. However, 30–45 % of patients continue to experience anemia and require replacement blood transfusions after 6 months or more of regular C5 inhibitor therapy. It has been established that the most common cause of a suboptimal treatment response is extravascular hemolysis, which is based on opsonization of PNH erythrocytes by C3b complement fragments followed by phagocytosis and degradation of these cells by macrophages in the liver and spleen. To suppress the mechanism of extravascular hemolysis, inhibitors of the proximal complement pathway have been developed, including the C3 inhibitor pegcetacoplan, which was registered in Russia in 2023 under the trade name Empaveli. The drug inhibits the activity of C3 and C3b complement components and thereby blocks the entire complement activation cascade, providing suppression of both intravascular and extravascular hemolysis. This article presents the first Russian experience of treating nine PNH patients with suboptimal response to C5 inhibitor therapy with pegcetacoplan. All nine patients demonstrated an average increase in hemoglobin levels of 25 g / L from baseline, achieving transfusion independence. The article also provides a detailed description of three clinical cases of particular interest.
